Relief For Sickle Cell Patients As Ghana Adopts Novartis hydroxyurea

Wednesday, November 6, 2019, marked the historic launch of the first biggest ever Public Private Partnership (PPP) initiative between the Government of Ghana and Global Pharmaceutical Giant, Novartis for the treatment of sickle cell disease in Ghana.

Through the partnership, Novartis’ hydroxyurea medication would be made available to sickle cell patients across Ghana.

The partnership will see the expansion of access to sickle cell medication and related treatment to people living with Sickle Cell Disease (SCD).

The PPP between government, pharmaceutical giant, Novartis, the Sickle Cell Foundation Ghana, Ghana Health Service and the Ministry of Health, will improve the diagnoses and accelerate treatment for people with SCD in the country with the introduction of hydroxyurea to the treatment line at affordable cost.

Specifically, the partners will collaborate on field testing and implementation of SCD treatment guidelines, the establishment of cross-regional centers of excellence and the implementation of newborn screening at these centers.

Dr. Bawumia, speaking at the event which brought together over 500 stakeholders in the health sector, said Ghana by the PPP will be the first African country to ensure that hydroxyurea is accessible to people living with SCD at affordable cost.

He said government was working to ensure costs of hydroxyurea is covered in the National Health Insurance Scheme (NHIS).

“Covering the cost of hydroxyurea for people with sickle cell disease in this country will bring much needed financial relief to families struggling with the cost of care for their loved ones with sickle cell disease,” he said.

The Vice president said, there are steps being taken to advance discussion for the acceleration of parliamentary budget approval of hydroxyurea coverage.

“Our goal collectively, is to aspire to a nation where children attend school without being stigmatized; where adults can be employed without fear of discrimination… In a nutshell we will make every effort to normalize sickle cell disease within the Ghanaian society, he said.

In Ghana, it is estimated that 15,000 babies are born with sickle cell disease every year out of which 90 per cent die before they are five years old due to preventable complications.

Hydroxyurea treatment is highly effective in treating SCD by reducing the incidence of vaso-occlusive event, infections, malaria, transfusion and death.

Health Minister, Kwaku Agyemang Manu, said the five-year agreement will focus on four key areas including treatment, diagnosis, research and advocacy to help reduce the burden of SCD.

He said already the Health Ministry has taken delivery of 20,000 free hydroxyurea medications with the number expected to increase to 60,000 by the end of the year.

The medications he said, will be distributed free of charge to patients with SCD who have registered with Sickle cell centers, particularly children under five years, by trained doctors in the various hospitals.

He further expressed the Ministry’s commitment to, ‘put SCD among the priorities on our national health agenda and to put the required resources behind it.’

CEO of Novartis, Dr. Vas Narasimhan, expressed his excitement at the partnership adding at Novartis will be developing a paediatric formulation of hydroxyurea to enable easy administration to babies.

“Novartis is deeply committed to reimagining medicine for patients in Ghana and across Africa,” said the CEO.

“Building on our long heritage of addressing malaria and leprosy in Africa, we’re excited to help improve the diagnosis and treatment of people with sickle cell disease here in Ghana. Novartis is proud to join this pioneering partnership, which could accelerate efforts to forge a healthier future for children across the continent.”

He encouraged new born screening of the SCD.

He further noted that Novartis will be scaling up the programme in other Sub Saharan African Countries.

“Novartis has a long-term commitment to ensuring that our medicines, and healthcare in general, are accessible to as many patients as possible.
Our hope is that we’ll continue to reimagine the way this disease is treated, in order to offer better medicines and improved care to sickle cell patients in Africa and around the world. I am proud that Novartis is committed to addressing this challenge,” he said.

To date, Novartis has delivered more than 20 000 treatments of hydroxyurea. Initially, the therapy will be made available through 11 trained treatment centers, as well as through private distribution channels, and is expected to cover the needs of patients for up to 12 months.

The partners aim to open more treatment centers by the end of the year, and Novartis is committed to delivering a total of 60 000 treatments.

Discussions are already underway for inclusion of the medicine and associated laboratory testing in the National Health Insurance Scheme, as well as prioritizing SCD as a national program.

Hydroxyurea is a commonly used medicine for patients with SCD in developed countries, and is approved for use in both adults and children.

In October 2018, the Ghana FDA granted marketing authorization to Novartis hydroxyurea, making it the first time that hydroxyurea would be available for this indication in Ghana.

A recent study published in the New England Journal of Medicine3 indicates that hydroxyurea treatment is effective and safe in children with SCD in sub-Saharan Africa and reduces the incidence of pain events (vaso-occlusive crises), malaria, blood transfusions, and death.

“All the traditional names by which SCD is known allude to the episodes of severe pain experienced by people with the disease,” said Prof. Kwaku Ohene-Frempong, MD, President of the Sickle Cell Foundation of Ghana and Program Coordinator for the National Newborn Screening Program for Sickle Cell Disease.

“Hydroxyurea is a drug with proven efficacy in reducing pain episodes and other complications of the disease.

Currently, the highest standard of care for people with SCD starts with early diagnosis through newborn screening followed by penicillin prophylaxis to prevent early death from infection, and hydroxyurea therapy to ease the pain, prevent other complications and improve quality of life.”

At the same time, Novartis has committed to develop a child-friendly formulation of hydroxyurea and has announced plans to conduct two clinical trials in Ghana and Kenya for its next-generation treatment for SCD, crizanlizumab.

Crizanlizumab is a novel targeted biologic therapy that is expected to help reduce pain crises in people with SCD.

The trials are expected to start in 2020; this will be the first time that a biologic therapy, which is not a vaccine, enters multicenter clinical trials in sub-Saharan Africa (excluding South Africa)4.

Sickle cell disease is recognized by the World Health Organization as a public health priority and a neglected health problem in sub-Saharan Africa.

Approximately 80% of individuals with
SCD globally are born in sub-Saharan Africa, and there is evidence to suggest that more than half of affected individuals may die before the age of five due to preventable complications.

In Ghana, it is estimated that 15 000 babies are born with sickle cell disease every year2.

The five-year partnership aims to improve and extend the lives of people with SCD through a comprehensive approach to screening and diagnosis; treatment and disease management; training and education; and elevating basic and clinical research capabilities. Specifically, the partners aim to collaborate on field-testing and implementation of SCD treatment guidelines, the establishment of centers of excellence across regions and the implementation of newborn screening at these centers. In addition, partners plan to employ digital technologies to monitor and evaluate patient registration, report real-time data and help ensure safe large-scale rollout of medicine.

In parallel, Novartis is working with Zipline, a California-based automated logistics company, to make sickle cell treatments widely available, especially in rural areas. Zipline is already operating two distribution centers in Ghana, in Omenako and Asante Mampong, with plans to open two more in the near future.

In addition, in order to help further ensure a sustainable supply of high quality hydroxyurea, Novartis has entered a strategic collaboration with Olon, a manufacturer based in Italy that supplies the active ingredient for the drug. “We are proud to collaborate with Novartis and its partners to help make hydroxyurea more broadly available and in a sustainable way,” said Paolo Tubertini, CEO of Olon. “As a demonstration of our commitment to patients with sickle cell disease and this program, we will also contribute, without cost, the active ingredient to cover approximately 12 000 thousand treatments. Our hope is to continue to meet the needs and challenges of those living with sickle cell disease and their families to improve access to quality health c

Blood vessels and blood cells become sticky due to damage caused by sickle cells and ongoing chronic inflammation 5,6.

That leads to blood cells sticking to each other and to the blood vessels and causing blockages – called vaso-occlusion – that can lead to the acute episodes of pain (known as sickle cell pain crises or vaso-occlusive crises), stroke and other life-threatening complications5,7.

It is a lifelong illness that can put an emotional, physical, and financial burden on patients and their families8,9.

SCD is a global health problem, with the highest burden of disease concentrated in sub-Saharan Africa.

In countries in West, Central and East Africa, the prevalence of the sickle cell gene is between 10 to 30 percent, while in some areas it is as high as 45%10. It is estimated that approximately 1 000 children in Africa are born with SCD every day and more than half die before they reach five years of age11.

This is due primarily to lack of early diagnosis through newborn screening, penicillin prophylaxis, parental education, and comprehensive care. In resource-poor countries, more than 90 percent of children with SCD do not survive to adulthood12.

Despite the adoption by the WHO of an SCD strategy for Africa in 2010, the disease is largely absent from the global or national health agenda.